Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

Official title:

Safety and Efficacy of Hetrombopag in Children and Adolescents With Chronic Primary Immune Thrombocytopenia：a Randomized, Multicenter, Placebo-controlled Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04737850
• Other study ID #: HR-TPO-ITP-III-PED
• Status: Recruiting
• Phase: Phase 3
• Start date: March 29, 2021
• Est. completion date: May 2023

Study information

• Verified date: August 2022
• Source: Jiangsu HengRui Medicine Co., Ltd.
• Contact: Tianyou Wang, Ph.D
• Phone: 010-59616161
• Email: wangtianyou[@]bch.com.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to investigate the efficacy, safety of Hetrombopag in children with previously treated chronic immune thrombocytopenia who are between 6 and 17 years of age. This is a 2 part study. In part A, patients will receive Hetrombopag for 8 weeks. In part B, all patients will receive Hetrombopag for 24 weeks.

Description:

This is a two-part, double-blind, randomized, placebo-controlled, and open-label Phase III study to investigate the efficacy, safety of Hetrombopag in pediatric patients with previously treated chronic ITP. In Part A, patients will receive Hetrombopag for 8 weeks. After completing Part A, patients will begin Part B, in which they will be randomized to receive Hetrombopag or placebo in a 12 week double-blind, placebo-controlled treatment period, following an open-label 12 week treatment period.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 117
• Est. completion date: May 2023
• Est. primary completion date: May 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 17 Years

Eligibility

Inclusion Criteria:

1. Age = 6 years old and = 17 years old,both sexes.

2. Part A:Confirmed diagnosis of ITP =6months; Part B: Confirmed diagnosis of ITP =12months;Platelets <30×10^9/L twice in a row,and platelets <30×10^9/L before taking the medicine.

3. Subjects who are refractory or have relapsed after at least one prior ITP therapy.

4. Birth control during and 28 days after the trial.

5. Written informed consent must be obtained from the patient's guardian and accompanying informed assent from the patient (for children over 8 years old).

Exclusion Criteria:

1. No evidence of other causes of thrombocytopenia.

2. Diagnosis as Evans or Wiskott-Aldrich comprehensive.

3. Patients with any prior history of arterial or venous thrombosis, or diagnosis as thrombophilia.

4. Suffering from serious, progressive, uncontrolled kidney, liver, gastrointestinal, endocrine, lung, heart, nervous system, brain, or mental illness.

5. ALT, AST, or ALP> 1.5 x upper limit of normal (ULN), DBLI, or Scr > 1.2 x upper limit of normal (ULN).

6. Active HIV or HCV-Ab positive,HBsAg positive.

7. PT result exceeds normal by more than ±3s, APTT result exceeds normal by more than ±10s

8. Participated in clinical trials of other drugs (received experimental drugs) within 3 months prior to medication.

9. The inestigators determined that other conditions were inappropriate for participation in this clinical trial.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Hetrombopag
Thrombopoietin receptor agonist
• Placebo
Placebo with no active pharmaceutical ingredient

Locations

Country	Name	City	State
China	Beijing children's hospital .Capital medical university	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Jiangsu HengRui Medicine Co., Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	the main parameters in population PK/PD modeling in Part A	Peak Plasma Concentration (Cmax)	from baseline to Week 2
Primary	the proportion of patients with a platelet count =50×10^9/L at week 10.	efficacy in part B	from baseline to Week 10